Novartis Inks $2.9 Billion Deal to Acquire Global Rights to PTC518, a Novel Oral Therapy for Huntington’s Disease

Dec 02, 2024 20:58 CST Updated 20:58

PTC Therapeutics

Innovative Small Molecule and Gene Therapy Drug Developer

Novartis

Drug Development and Manufacturing

December 2,Warren, New Jersey – PTC Therapeutics (NASDAQ: PTCT) announced an exclusive global licensing and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis, for the development of PTC518 in treating Huntington's disease indications, including related molecules. Under the agreement, PTC will receive an upfront payment of $1 billion, up to $1.9 billion in development, regulatory, and sales milestones, a profit share in the United States, and tiered double-digit royalties on sales outside the U.S. The U.S. FDA is scheduled to meet in the fourth quarter of 2024 to discuss.PTC518Accelerated Approval Pathway.

Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, stated:"PTC518 is the leading oral disease-modifying therapy in development for Huntington's disease, and the economics of this agreement align with the promise of this treatment. This collaboration combines PTC's expertise in developing small molecule splicing therapies with Novartis' global experience in neuroscience therapy development and commercialization. We are excited to partner with Novartis to accelerate the potential of PTC518, bringing treatment to hundreds of thousands of HD patients worldwide who need well-tolerated and effective disease-modifying therapies. PTC will use the proceeds from this transaction to expand our splicing platform and support commercial and development portfolio activities."

Novartis CEO Vas Narasimhan said:"Huntington's disease is a devastating and fatal hereditary disorder. This agreement with PTC aims to strengthen our neuroscience pipeline and reflects our strategic focus and commitment to exploring new, potentially transformative approaches to treat neurodegenerative diseases with unmet needs. We look forward to leveraging our expertise in neurodegenerative diseases and experience in HD to advance this potential first-in-class oral treatment for the HD community."

PTC518 was discovered from PTC’s validated splicing platform and is currently undergoing the Phase 2 PIVOT-HD clinical trial. Interim results reported in June 2024 indicated that PTC518 treatment led to a sustained, dose-dependent reduction in mutant huntingtin protein (HTT) levels in both blood and cerebrospinal fluid (CSF), with early signals of dose-dependent benefits in key clinical measures at 12 months. Importantly, PTC518 continues to demonstrate a favorable safety and tolerability profile.

After the completion of the placebo-controlled portion of PIVOT-HD, Novartis will be responsible for the development, manufacturing, and commercialization of PTC518, with completion expected in the first half of 2025.The two companies will share profits and losses in the United States in a 40/60 ratio (40% for PTC and 60% for Novartis).The completion of the transaction is subject to customary closing conditions, including regulatory approvals.The two parties expect the agreement to be completed in the first quarter of 2025.

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